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The Amicus Therapeutics and Their Migalastat Monotherapy Method

While the healthcare industry is moving forward and opening the path for new methods and treatments to a varied number of diseases, the practice of developing new ways of executing therapies is one of the most significant assets for the progression of medicine as a whole.

A famous therapeutic institute located in New Jersy, Cranbury, is contributing to the improvement of this front since their foundation in February 4, 2002. Amicus Therapeutics was created with the intention of focusing their research on rare and orphan diseases that have unique peculiarities and deserve a higher attention from some of the best physicians and researchers in the United States.

These disorders that Amicus Therapeutics focuses on are called “Lysosomal Storage Disorders” and are very detrimental to some individuals who suffer from a number of these and have very limited assistance from researches. Around the world, there are diseases that have a higher percentage of institutions taking care of discovering new methods of treatment and therapies for more famous diseases like Aids, cancer, and others. The Lysosomal Disorders don’t have enough backup support from physicians of skill and experience. Thus Amicus Therapeutics is aiming to change this reality for the future.

The company has their focus on the Chaperone-Advanced Replacement Therapy, a method that has the replacement of enzymes in the body of the infected, and were one of the pioneers to develop a more reliable and effective version of the therapy. Amicus Therapeutics was also a company that went public five years after their inauguration, which means that anyone can fund and support the institution and helps progress their therapy development. The clinic and their professional staff are dedicating their efforts into proceeding with the migalastat monotherapy, one of their greatest projects for the future.

The enterprise is aiming to stabilize what they call “alpha-galactosidase,” an endogenous mutant cell that is responsible for the type of disorder that the group treats and develops therapies for. Amicus Therapeutics currently has no established products in the market (Google Finance), and instead focuses on the sole purpose of supporting the investigation and building collaborations with other important corporations to try and further their reach.

Amicus Therapeutics, contrary to popular belief, is not centered only in New Jersey, where they are headquartered, but also has a subsidiary in San Diego since 2008, one year after the group went public. The researches and therapy development of the group is not alone, as they have the support and funding of many important personalities and institutions for the healthcare department, like the 2010 $500,000 fund from Michael J. Fox Foundation.